{"id":26340,"date":"2023-04-22T22:51:32","date_gmt":"2023-04-22T15:51:32","guid":{"rendered":"http:\/\/noithathoaphat.in\/ctv\/?p=26340"},"modified":"2023-04-22T22:51:32","modified_gmt":"2023-04-22T15:51:32","slug":"trofinetide-phuong-phap-dieu-tri-dau-tien-cho-hoi-chung-rett","status":"publish","type":"post","link":"http:\/\/noithathoaphat.in\/ctv\/index.php\/2023\/04\/22\/trofinetide-phuong-phap-dieu-tri-dau-tien-cho-hoi-chung-rett\/","title":{"rendered":"Trofinetide – Ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean cho h\u1ed9i ch\u1ee9ng Rett"},"content":{"rendered":"

Trofinetide l\u00e0 m\u1ed9t lo\u1ea1i thu\u1ed1c \u0111ang \u0111\u01b0\u1ee3c nghi\u00ean c\u1ee9u v\u00e0 s\u1eed d\u1ee5ng trong \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Rett, m\u1ed9t b\u1ec7nh hi\u1ebfm li\u00ean quan \u0111\u1ebfn s\u1ef1 ph\u00e1t tri\u1ec3n n\u00e3o b\u1ed9 \u1edf tr\u1ebb em g\u00e1i. \u0110\u00e2y l\u00e0 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t cho b\u1ec7nh n\u00e0y v\u00e0 \u0111\u00e3 \u0111\u01b0\u1ee3c FDA (C\u1ee5c qu\u1ea3n l\u00fd Th\u1ef1c ph\u1ea9m v\u00e0 D\u01b0\u1ee3c ph\u1ea9m Hoa K\u1ef3) ch\u1ea5p thu\u1eadn s\u1eed d\u1ee5ng. Trofinetide \u0111\u01b0\u1ee3c cho l\u00e0 c\u00f3 kh\u1ea3 n\u0103ng c\u1ea3i thi\u1ec7n c\u00e1c tri\u1ec7u ch\u1ee9ng c\u1ee7a b\u1ec7nh Rett nh\u01b0 gi\u1ea3m s\u1ef1 suy gi\u1ea3m ch\u1ee9c n\u0103ng n\u00e3o b\u1ed9, gi\u1ea3m b\u1ec7nh co gi\u1eadt v\u00e0 c\u1ea3i thi\u1ec7n kh\u1ea3 n\u0103ng giao ti\u1ebfp c\u1ee7a b\u1ec7nh nh\u00e2n<\/strong><\/p>\n

1.T\u1ed5ng quan v\u1ec1 h\u1ed9i ch\u1ee9ng Rett<\/strong><\/h2>\n

1.1 H\u1ed9i ch\u1ee9ng Rett l\u00e0 g\u00ec?<\/span><\/h3>\n

H\u1ed9i ch\u1ee9ng Rett l\u00e0 m\u1ed9t r\u1ed1i lo\u1ea1n ph\u00e1t tri\u1ec3n v\u00e0 th\u1ea7n kinh di truy\u1ec1n hi\u1ebfm g\u1eb7p, \u1ea3nh h\u01b0\u1edfng \u0111\u1ebfn c\u00e1ch th\u1ee9c ph\u00e1t tri\u1ec3n c\u1ee7a n\u00e3o b\u1ed9. R\u1ed1i lo\u1ea1n n\u00e0y g\u00e2y ra s\u1ef1 m\u1ea5t d\u1ea7n c\u00e1c k\u1ef9 n\u0103ng v\u1eadn \u0111\u1ed9ng v\u00e0 ng\u00f4n ng\u1eef. H\u1ed9i ch\u1ee9ng Rett ch\u1ee7 y\u1ebfu \u1ea3nh h\u01b0\u1edfng \u0111\u1ebfn ph\u1ee5 n\u1eef. H\u1ea7u h\u1ebft tr\u1ebb s\u01a1 sinh m\u1eafc h\u1ed9i ch\u1ee9ng Rett d\u01b0\u1eddng nh\u01b0 ph\u00e1t tri\u1ec3n b\u00ecnh th\u01b0\u1eddng trong 6 th\u00e1ng \u0111\u1ea7u \u0111\u1eddi, sau \u0111\u00f3 m\u1ea5t \u0111i c\u00e1c k\u1ef9 n\u0103ng \u0111\u00e3 c\u00f3 tr\u01b0\u1edbc \u0111\u00e2y (b\u00f2, giao ti\u1ebfp, s\u1eed d\u1ee5ng tay,…). Theo th\u1eddi gian, tr\u1ebb m\u1eafc h\u1ed9i ch\u1ee9ng Rett ng\u00e0y c\u00e0ng g\u1eb7p nhi\u1ec1u v\u1ea5n \u0111\u1ec1 v\u1edbi vi\u1ec7c s\u1eed d\u1ee5ng c\u00e1c c\u01a1 ki\u1ec3m so\u00e1t chuy\u1ec3n \u0111\u1ed9ng, ph\u1ed1i h\u1ee3p v\u00e0 giao ti\u1ebfp. H\u1ed9i ch\u1ee9ng Rett c\u0169ng c\u00f3 th\u1ec3 g\u00e2y co gi\u1eadt v\u00e0 thi\u1ec3u n\u0103ng tr\u00ed tu\u1ec7<\/span><\/p>\n

\"H\u1ed9i
H\u1ed9i ch\u1ee9ng Rett<\/figcaption><\/figure>\n

1.2 Nguy\u00ean nh\u00e2n g\u00e2y ra h\u1ed9i ch\u1ee9ng Rett?<\/span><\/h3>\n

H\u1ed9i ch\u1ee9ng Rett l\u00e0 m\u1ed9t r\u1ed1i lo\u1ea1n di truy\u1ec1n hi\u1ebfm g\u1eb7p. H\u1ed9i ch\u1ee9ng Rett c\u1ed5 \u0111i\u1ec3n, c\u0169ng nh\u01b0 m\u1ed9t s\u1ed1 bi\u1ebfn th\u1ec3 (h\u1ed9i ch\u1ee9ng Rett kh\u00f4ng \u0111i\u1ec3n h\u00ecnh) v\u1edbi c\u00e1c tri\u1ec7u ch\u1ee9ng nh\u1eb9 h\u01a1n ho\u1eb7c nghi\u00eam tr\u1ecdng h\u01a1n, x\u1ea3y ra d\u1ef1a tr\u00ean m\u1ed9t s\u1ed1 thay \u0111\u1ed5i di truy\u1ec1n (\u0111\u1ed9t bi\u1ebfn). Nh\u1eefng thay \u0111\u1ed5i di truy\u1ec1n g\u00e2y ra h\u1ed9i ch\u1ee9ng Rett x\u1ea3y ra ng\u1eabu nhi\u00ean, th\u01b0\u1eddng l\u00e0 \u1edf gen MECP2<\/a><\/span><\/p>\n

1.3 Tri\u1ec7u ch\u1ee9ng khi m\u1eafc ph\u1ea3i h\u1ed9i ch\u1ee9ng Rett?<\/span><\/h3>\n

C\u00e1c d\u1ea5u hi\u1ec7u v\u00e0 tri\u1ec7u ch\u1ee9ng ch\u00ednh bao g\u1ed3m<\/span><\/p>\n

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  • T\u0103ng tr\u01b0\u1edfng ch\u1eadm l\u1ea1i: S\u1ef1 ph\u00e1t tri\u1ec3n c\u1ee7a n\u00e3o ch\u1eadm l\u1ea1i sau khi sinh. K\u00edch th\u01b0\u1edbc \u0111\u1ea7u nh\u1ecf h\u01a1n b\u00ecnh th\u01b0\u1eddng (ch\u1ee9ng \u0111\u1ea7u nh\u1ecf) \u0111\u00f4i khi l\u00e0 d\u1ea5u hi\u1ec7u \u0111\u1ea7u ti\u00ean cho th\u1ea5y tr\u1ebb m\u1eafc h\u1ed9i ch\u1ee9ng Rett. Khi tr\u01b0\u1edfng th\u00e0nh, c\u00e1c b\u1ed9 ph\u1eadn kh\u00e1c c\u1ee7a c\u01a1 th\u1ec3 ch\u1eadm ph\u00e1t tri\u1ec3n<\/span><\/li>\n
  • M\u1ea5t kh\u1ea3 n\u0103ng v\u1eadn \u0111\u1ed9ng v\u00e0 ph\u1ed1i h\u1ee3p: C\u00e1c d\u1ea5u hi\u1ec7u \u0111\u1ea7u ti\u00ean th\u01b0\u1eddng bao g\u1ed3m gi\u1ea3m ki\u1ec3m so\u00e1t tay v\u00e0 gi\u1ea3m kh\u1ea3 n\u0103ng b\u00f2 ho\u1eb7c \u0111i b\u1ed9. L\u00fac \u0111\u1ea7u, s\u1ef1 m\u1ea5t kh\u1ea3 n\u0103ng n\u00e0y di\u1ec5n ra nhanh ch\u00f3ng, sau \u0111\u00f3 n\u00f3 ti\u1ebfp t\u1ee5c di\u1ec5n ra d\u1ea7n d\u1ea7n. Cu\u1ed1i c\u00f9ng, c\u00e1c c\u01a1 tr\u1edf n\u00ean y\u1ebfu ho\u1eb7c c\u1ee9ng, v\u1edbi chuy\u1ec3n \u0111\u1ed9ng v\u00e0 v\u1ecb tr\u00ed b\u1ea5t th\u01b0\u1eddng<\/span><\/li>\n
  • M\u1ea5t kh\u1ea3 n\u0103ng giao ti\u1ebfp: Tr\u1ebb m\u1eafc h\u1ed9i ch\u1ee9ng Rett th\u01b0\u1eddng b\u1eaft \u0111\u1ea7u m\u1ea5t kh\u1ea3 n\u0103ng n\u00f3i, giao ti\u1ebfp b\u1eb1ng m\u1eaft v\u00e0 giao ti\u1ebfp theo nh\u1eefng c\u00e1ch kh\u00e1c. M\u1ed9t s\u1ed1 tr\u1ebb c\u00f3 nh\u1eefng thay \u0111\u1ed5i nhanh ch\u00f3ng, ch\u1eb3ng h\u1ea1n nh\u01b0 m\u1ea5t ng\u00f4n ng\u1eef \u0111\u1ed9t ng\u1ed9t. Theo th\u1eddi gian, tr\u1ebb c\u00f3 th\u1ec3 d\u1ea7n l\u1ea5y l\u1ea1i giao ti\u1ebfp b\u1eb1ng m\u1eaft v\u00e0 ph\u00e1t tri\u1ec3n c\u00e1c k\u1ef9 n\u0103ng giao ti\u1ebfp phi ng\u00f4n ng\u1eef<\/span><\/li>\n
  • Chuy\u1ec3n \u0111\u1ed9ng tay b\u1ea5t th\u01b0\u1eddng: Tr\u1ebb m\u1eafc h\u1ed9i ch\u1ee9ng Rett th\u01b0\u1eddng ph\u00e1t tri\u1ec3n c\u00e1c c\u1eed \u0111\u1ed9ng tay l\u1eb7p \u0111i l\u1eb7p l\u1ea1i, kh\u00f4ng c\u00f3 m\u1ee5c \u0111\u00edch, kh\u00e1c nhau \u1edf m\u1ed7i tr\u1ebb. C\u00e1c chuy\u1ec3n \u0111\u1ed9ng c\u1ee7a tay c\u00f3 th\u1ec3 bao g\u1ed3m v\u1eb7n, b\u00f3p, v\u1ed7, g\u00f5 ho\u1eb7c xoa tay<\/span><\/li>\n<\/ul>\n

    C\u00e1c d\u1ea5u hi\u1ec7u v\u00e0 tri\u1ec7u ch\u1ee9ng kh\u00e1c nh\u01b0:\u00a0<\/span><\/p>\n

      \n
    • Chuy\u1ec3n \u0111\u1ed9ng m\u1eaft b\u1ea5t th\u01b0\u1eddng: Tr\u1ebb m\u1eafc h\u1ed9i ch\u1ee9ng Rett c\u00f3 xu h\u01b0\u1edbng c\u1eed \u0111\u1ed9ng m\u1eaft b\u1ea5t th\u01b0\u1eddng, ch\u1eb3ng h\u1ea1n nh\u01b0 nh\u00ecn ch\u1eb1m ch\u1eb1m, ch\u1edbp m\u1eaft, l\u00e1c m\u1eaft ho\u1eb7c nh\u1eafm m\u1ed9t m\u1eaft<\/span><\/li>\n
    • C\u00e1c v\u1ea5n \u0111\u1ec1 v\u1ec1 h\u00f4 h\u1ea5p: N\u00edn th\u1edf, th\u1edf nhanh (t\u0103ng th\u00f4ng kh\u00ed), th\u1ed5i m\u1ea1nh kh\u00f4ng kh\u00ed ho\u1eb7c n\u01b0\u1edbc b\u1ecdt v\u00e0 nu\u1ed1t kh\u00f4ng kh\u00ed. C\u00e1c r\u1ed1i lo\u1ea1n h\u00f4 h\u1ea5p kh\u00e1c nh\u01b0 th\u1edf n\u00f4ng ho\u1eb7c ng\u1eebng th\u1edf trong th\u1eddi gian ng\u1eafn (ng\u01b0ng th\u1edf) c\u00f3 th\u1ec3 x\u1ea3y ra trong khi ng\u1ee7<\/span><\/li>\n
    • Kh\u00f3 ch\u1ecbu v\u00e0 kh\u00f3c. Tr\u1ebb m\u1eafc h\u1ed9i ch\u1ee9ng Rett c\u00f3 th\u1ec3 ng\u00e0y c\u00e0ng tr\u1edf n\u00ean k\u00edch \u0111\u1ed9ng v\u00e0 c\u00e1u k\u1ec9nh khi l\u1edbn l\u00ean. Nh\u1eefng kho\u1ea3ng th\u1eddi gian kh\u00f3c ho\u1eb7c la h\u00e9t c\u00f3 th\u1ec3 b\u1eaft \u0111\u1ea7u \u0111\u1ed9t ng\u1ed9t, kh\u00f4ng c\u00f3 l\u00fd do r\u00f5 r\u00e0ng v\u00e0 k\u00e9o d\u00e0i h\u00e0ng gi\u1edd. M\u1ed9t s\u1ed1 tr\u1ebb c\u00f3 th\u1ec3 c\u1ea3m th\u1ea5y s\u1ee3 h\u00e3i v\u00e0 lo l\u1eafng<\/span><\/li>\n
    • Thi\u1ec3u n\u0103ng tr\u00ed tu\u1ec7. M\u1ea5t k\u1ef9 n\u0103ng c\u00f3 th\u1ec3 li\u00ean quan \u0111\u1ebfn vi\u1ec7c m\u1ea5t kh\u1ea3 n\u0103ng suy ngh\u0129, hi\u1ec3u v\u00e0 h\u1ecdc h\u1ecfi.<\/span><\/li>\n<\/ul>\n

      1.4 Di\u1ec5n bi\u1ebfn c\u1ee7a h\u1ed9i ch\u1ee9ng Rett?<\/span><\/h3>\n

      H\u1ed9i ch\u1ee9ng Rett th\u01b0\u1eddng \u0111\u01b0\u1ee3c chia th\u00e0nh b\u1ed1n giai \u0111o\u1ea1n:<\/span><\/p>\n

      Giai \u0111o\u1ea1n 1: Kh\u1edfi ph\u00e1t s\u1edbm\u00a0<\/span><\/p>\n

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      • C\u00e1c d\u1ea5u hi\u1ec7u v\u00e0 tri\u1ec7u ch\u1ee9ng r\u1ea5t tinh t\u1ebf v\u00e0 d\u1ec5 b\u1ecb b\u1ecf qua trong giai \u0111o\u1ea1n \u0111\u1ea7u ti\u00ean, b\u1eaft \u0111\u1ea7u t\u1eeb 6 \u0111\u1ebfn 18 th\u00e1ng tu\u1ed5i. Giai \u0111o\u1ea1n 1 c\u00f3 th\u1ec3 k\u00e9o d\u00e0i v\u00e0i th\u00e1ng ho\u1eb7c m\u1ed9t n\u0103m. Tr\u1ebb trong giai \u0111o\u1ea1n n\u00e0y c\u00f3 th\u1ec3 \u00edt giao ti\u1ebfp b\u1eb1ng m\u1eaft h\u01a1n v\u00e0 b\u1eaft \u0111\u1ea7u m\u1ea5t h\u1ee9ng th\u00fa v\u1edbi \u0111\u1ed3 ch\u01a1i, c\u0169ng c\u00f3 th\u1ec3 ch\u1eadm ng\u1ed3i ho\u1eb7c b\u00f2<\/span><\/li>\n<\/ul>\n

        Giai \u0111o\u1ea1n 2: Suy tho\u00e1i nhanh ch\u00f3ng<\/span><\/p>\n

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        • B\u1eaft \u0111\u1ea7u t\u1eeb 1 \u0111\u1ebfn 4 tu\u1ed5i, tr\u1ebb m\u1ea5t kh\u1ea3 n\u0103ng th\u1ef1c hi\u1ec7n c\u00e1c k\u1ef9 n\u0103ng \u0111\u00e3 c\u00f3 tr\u01b0\u1edbc \u0111\u00e2y. Di\u1ec5n ti\u1ebfn giai \u0111o\u1ea1n n\u00e0y di\u1ec5n ra nhanh ch\u00f3ng ho\u1eb7c d\u1ea7n d\u1ea7n, x\u1ea3y ra trong nhi\u1ec1u tu\u1ea7n ho\u1eb7c nhi\u1ec1u th\u00e1ng. C\u00e1c tri\u1ec7u ch\u1ee9ng c\u1ee7a h\u1ed9i ch\u1ee9ng Rett x\u1ea3y ra, ch\u1eb3ng h\u1ea1n nh\u01b0 ch\u1eadm ph\u00e1t tri\u1ec3n \u0111\u1ea7u, c\u1eed \u0111\u1ed9ng tay b\u1ea5t th\u01b0\u1eddng, th\u1edf nhanh, la h\u00e9t ho\u1eb7c kh\u00f3c m\u00e0 kh\u00f4ng c\u00f3 l\u00fd do r\u00f5 r\u00e0ng, c\u00e1c v\u1ea5n \u0111\u1ec1 v\u1ec1 c\u1eed \u0111\u1ed9ng v\u00e0 ph\u1ed1i h\u1ee3p, c\u0169ng nh\u01b0 m\u1ea5t kh\u1ea3 n\u0103ng giao ti\u1ebfp v\u00e0 t\u01b0\u01a1ng t\u00e1c x\u00e3 h\u1ed9i<\/span><\/li>\n<\/ul>\n

          Giai \u0111o\u1ea1n 3: \u0110\u1ec9nh \u0111i\u1ec3m\u00a0<\/span><\/p>\n

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          • Giai \u0111o\u1ea1n n\u00e0y th\u01b0\u1eddng b\u1eaft \u0111\u1ea7u t\u1eeb 2 \u0111\u1ebfn 10 tu\u1ed5i v\u00e0 c\u00f3 th\u1ec3 k\u00e9o d\u00e0i nhi\u1ec1u n\u0103m. M\u1eb7c d\u00f9 c\u00e1c v\u1ea5n \u0111\u1ec1 v\u1ec1 v\u1eadn \u0111\u1ed9ng v\u1eabn ti\u1ebfp di\u1ec5n, nh\u01b0ng h\u00e0nh vi c\u00f3 th\u1ec3 c\u1ea3i thi\u1ec7n \u0111\u00f4i ch\u00fat, \u00edt qu\u1ea5y kh\u00f3c v\u00e0 c\u00e1u k\u1ec9nh h\u01a1n, \u0111\u1ed3ng th\u1eddi c\u00f3 th\u1ec3 c\u00f3 m\u1ed9t s\u1ed1 c\u1ea3i thi\u1ec7n trong vi\u1ec7c s\u1eed d\u1ee5ng tay v\u00e0 giao ti\u1ebfp. Co gi\u1eadt c\u00f3 th\u1ec3 b\u1eaft \u0111\u1ea7u trong giai \u0111o\u1ea1n n\u00e0y v\u00e0 th\u01b0\u1eddng kh\u00f4ng x\u1ea3y ra tr\u01b0\u1edbc 2 tu\u1ed5i<\/span><\/li>\n<\/ul>\n

            Giai \u0111o\u1ea1n 4: Suy tho\u00e1i v\u1eadn \u0111\u1ed9ng mu\u1ed9n<\/span><\/p>\n

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            • Giai \u0111o\u1ea1n n\u00e0y th\u01b0\u1eddng b\u1eaft \u0111\u1ea7u sau 10 tu\u1ed5i v\u00e0 c\u00f3 th\u1ec3 k\u00e9o d\u00e0i h\u00e0ng n\u0103m ho\u1eb7c h\u00e0ng ch\u1ee5c n\u0103m. Trong giai \u0111o\u1ea1n n\u00e0y x\u1ea3y ra c\u00e1c d\u1ea5u hi\u1ec7u gi\u1ea3m kh\u1ea3 n\u0103ng v\u1eadn \u0111\u1ed9ng, y\u1ebfu c\u01a1, co r\u00fat kh\u1edbp v\u00e0 v\u1eb9o c\u1ed9t s\u1ed1ng. Kh\u1ea3 n\u0103ng hi\u1ec3u, giao ti\u1ebfp v\u00e0 k\u1ef9 n\u0103ng tay nh\u00ecn chung v\u1eabn \u1ed5n \u0111\u1ecbnh ho\u1eb7c c\u1ea3i thi\u1ec7n \u0111\u00f4i ch\u00fat v\u00e0 c\u00e1c c\u01a1n co gi\u1eadt c\u00f3 th\u1ec3 \u00edt x\u1ea3y ra h\u01a1n<\/span><\/li>\n<\/ul>\n

              1.5 C\u00e1c bi\u1ebfn ch\u1ee9ng c\u1ee7a h\u1ed9i ch\u1ee9ng Rett l\u00e0 g\u00ec?<\/span><\/h3>\n

              \u0102n u\u1ed1ng kh\u00f3 kh\u0103n, d\u1eabn \u0111\u1ebfn t\u00ecnh tr\u1ea1ng k\u00e9m dinh d\u01b0\u1ee1ng, ch\u1eadm l\u1edbn<\/span><\/p>\n

              C\u00e1c v\u1ea5n \u0111\u1ec1 v\u1ec1 c\u01a1, x\u01b0\u01a1ng, kh\u1edbp<\/span><\/p>\n

              Tu\u1ed5i th\u1ecd b\u1ecb r\u00fat ng\u1eafn<\/span><\/p>\n

              C\u00e1c v\u1ea5n \u0111\u1ec1 v\u1ec1 gi\u1ea5c ng\u1ee7<\/span><\/p>\n

              \u0110au c\u00f3 th\u1ec3 \u0111i k\u00e8m v\u1edbi c\u00e1c v\u1ea5n \u0111\u1ec1 nh\u01b0 c\u00e1c v\u1ea5n \u0111\u1ec1 v\u1ec1 \u0111\u01b0\u1eddng ti\u00eau h\u00f3a ho\u1eb7c g\u00e3y x\u01b0\u01a1ng.<\/span><\/p>\n

              1.6 Ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb\u00a0<\/span><\/h3>\n

              Cho \u0111\u1ebfn \u0111\u1ea7u n\u0103m 2023, ch\u01b0a c\u00f3 thu\u1ed1c \u0111\u1eb7c tr\u1ecb n\u00e0o \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t v\u00e0 ch\u1ea5p thu\u1eadn \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Rett, ch\u1ec9 c\u00f3 nh\u1eefng ph\u01b0\u01a1ng ph\u00e1p c\u1ea3i thi\u1ec7n v\u00e0 l\u00e0m gi\u1ea3m tri\u1ec7u ch\u1ee9ng nh\u01b0: v\u1eadt l\u00fd tr\u1ecb li\u1ec7u, h\u1ed7 tr\u1ee3 th\u1ec3 ch\u1ea5t, b\u1ed5 sung ch\u1ea5t dinh d\u01b0\u1ee1ng trong ch\u1ebf \u0111\u1ed9 \u0103n,…<\/span><\/p>\n

              2.Th\u00f4ng tin v\u1ec1 Trofinetide \u2013 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean cho h\u1ed9i ch\u1ee9ng Rett<\/strong><\/h2>\n

              V\u00e0o \u0111\u1ea7u th\u00e1ng 3 n\u0103m 2023, FDA \u0111\u00e3 ph\u00ea duy\u1ec7t Trofinetide d\u01b0\u1edbi d\u1ea1ng bi\u1ec7t d\u01b0\u1ee3c Daybue \u0111\u1ec3 \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Rett \u1edf b\u1ec7nh nh\u00e2n ng\u01b0\u1eddi l\u1edbn v\u00e0 tr\u1ebb em t\u1eeb 2 tu\u1ed5i tr\u1edf l\u00ean. \u0110\u00e2y l\u00e0 li\u1ec7u ph\u00e1p \u0111\u1ea7u ti\u00ean v\u00e0 duy nh\u1ea5t \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t cho h\u1ed9i ch\u1ee9ng Rett, \u0111\u00e1nh d\u1ea5u m\u1ed9t b\u01b0\u1edbc \u0111\u1ed9t ph\u00e1 l\u1edbn. Nghi\u00ean c\u1ee9u giai \u0111o\u1ea1n 3 LAVENDER\u00a0 \u0111\u00e3 ch\u1ee9ng minh s\u1ef1 c\u1ea3i thi\u1ec7n \u0111\u00e1ng k\u1ec3 v\u1ec1 m\u1eb7t th\u1ed1ng k\u00ea so v\u1edbi gi\u1ea3 d\u01b0\u1ee3c tr\u00ean c\u1ea3 hai ti\u00eau ch\u00ed \u0111\u00e1nh gi\u00e1 hi\u1ec7u qu\u1ea3. Nghi\u00ean c\u1ee9u giai \u0111o\u1ea1n 3 bao g\u1ed3m t\u1ed5ng c\u1ed9ng 187 b\u1ec7nh nh\u00e2n n\u1eef m\u1eafc h\u1ed9i ch\u1ee9ng Rett trong \u0111\u1ed9 tu\u1ed5i t\u1eeb 5 \u0111\u1ebfn 20 \u0111\u01b0\u1ee3c \u0111\u00e1nh gi\u00e1 trong kho\u1ea3ng th\u1eddi gian 12 tu\u1ea7n. K\u1ebft qu\u1ea3 th\u1eed nghi\u1ec7m cho th\u1ea5y Daybue hi\u1ec7n mang \u0111\u1ebfn kh\u1ea3 n\u0103ng t\u1ea1o ra s\u1ef1 kh\u00e1c bi\u1ec7t c\u00f3 \u00fd ngh\u0129a trong cu\u1ed9c s\u1ed1ng c\u1ee7a nhi\u1ec1u b\u1ec7nh nh\u00e2n v\u00ec t\u00ednh hi\u1ec7u qu\u1ea3 \u0111\u00fang nh\u01b0 mong \u0111\u1ee3i c\u1ee7a n\u00f3<\/span><\/p>\n

              \"Daybue
              Daybue (Trofinetide)<\/figcaption><\/figure>\n

              2.1 Nh\u00f3m d\u01b0\u1ee3c l\u00fd v\u00e0 d\u01b0\u1ee3c l\u1ef1c h\u1ecdc c\u1ee7a Trofinetide<\/span><\/h3>\n

              Trofinetide l\u00e0 m\u1ed9t ch\u1ea5t t\u01b0\u01a1ng t\u1ef1 t\u1ed5ng h\u1ee3p m\u1edbi c\u1ee7a Glypromate (Glycine-Proline-Glutamate \u2013 GPE), m\u1ed9t lo\u1ea1i protein xu\u1ea5t hi\u1ec7n t\u1ef1 nhi\u00ean trong n\u00e3o v\u00e0 tripeptide \u0111\u1ea7u t\u1eadn c\u00f9ng N c\u1ee7a y\u1ebfu t\u1ed1 t\u0103ng tr\u01b0\u1edfng gi\u1ed1ng insulin 1 (IGF-1). GPE th\u1ec3 hi\u1ec7n c\u00e1c \u0111\u1eb7c t\u00ednh b\u1ea3o v\u1ec7 th\u1ea7n kinh: c\u00e1c t\u1ebf b\u00e0o th\u1ea7n kinh \u0111\u01b0\u1ee3c b\u1ea3o v\u1ec7 kh\u1ecfi \u0111\u1ed9c t\u00ednh k\u00edch th\u00edch qua trung gian Glutamate v\u00e0 stress oxy h\u00f3a. V\u1edbi th\u1eddi gian b\u00e1n h\u1ee7y d\u00e0i h\u01a1n GPE, Trofinetide c\u0169ng c\u00f3 \u0111\u1eb7c t\u00ednh b\u1ea3o v\u1ec7 th\u1ea7n kinh t\u01b0\u01a1ng t\u1ef1\u00a0<\/span><\/p>\n

              2.2 C\u01a1 ch\u1ebf ho\u1ea1t \u0111\u1ed9ng c\u1ee7a Trofinetide<\/span><\/h3>\n

              H\u1ea7u h\u1ebft c\u00e1c tr\u01b0\u1eddng h\u1ee3p m\u1eafc h\u1ed9i ch\u1ee9ng Rett \u0111\u1ec1u li\u00ean quan \u0111\u1ebfn \u0111\u1ed9t bi\u1ebfn m\u1ea5t ch\u1ee9c n\u0103ng trong protein li\u00ean k\u1ebft methyl CpG m\u00e3 h\u00f3a gen 2 (MECP2), m\u1ed9t protein li\u00ean k\u1ebft DNA c\u00f3 vai tr\u00f2 \u0111i\u1ec1u h\u00f2a bi\u1ec3u hi\u1ec7n c\u1ee7a gen. Nh\u1eefng \u0111\u1ed9t bi\u1ebfn n\u00e0y \u0111\u01b0\u1ee3c cho l\u00e0 d\u1eabn \u0111\u1ebfn s\u1ef1 kh\u00f4ng tr\u01b0\u1edfng th\u00e0nh c\u1ee7a kh\u1edbp th\u1ea7n kinh \u1edf v\u1ecf n\u00e3o, s\u1ef1 chuy\u1ec3n h\u00f3a b\u1ea5t th\u01b0\u1eddng c\u1ee7a Cholesterol trong n\u00e3o, t\u1eeb \u0111\u00f3 d\u1eabn \u0111\u1ebfn s\u1ef1 ph\u00e1t tri\u1ec3n t\u1ebf b\u00e0o th\u1ea7n kinh b\u1ea5t th\u01b0\u1eddng v\u00e0 t\u00edn hi\u1ec7u th\u1ea7n kinh b\u1ea5t th\u01b0\u1eddng. Trofinetide \u0111\u00e3 c\u1ea3i thi\u1ec7n ch\u1ee9c n\u0103ng v\u1eadn \u0111\u1ed9ng v\u00e0 h\u00f4 h\u1ea5p c\u1ee7a tim, t\u0103ng tr\u1ecdng l\u01b0\u1ee3ng n\u00e3o v\u00e0 k\u00e9o d\u00e0i tu\u1ed5i th\u1ecd khi thi\u1ebfu MECP2. Ngo\u00e0i ra, Trofinetide t\u01b0\u01a1ng t\u1ef1 v\u1edbi GPE khi l\u00e0m gi\u1ea3m qu\u00e1 tr\u00ecnh ch\u1ebft theo ch\u01b0\u01a1ng tr\u00ecnh,\u00a0 gi\u1ea3m vi\u00eam, t\u1ed5n th\u01b0\u01a1ng m\u00f4 do \u0111\u1ed9c t\u00ednh k\u00edch th\u00edch do \u0111\u00f3 b\u1ea3o v\u1ec7 c\u00e1c t\u1ebf b\u00e0o th\u1ea7n kinh<\/span><\/p>\n

              2.3 D\u01b0\u1ee3c \u0111\u1ed9ng h\u1ecdc\u00a0<\/span><\/h3>\n

              H\u1ea5p thu: T max l\u00e0 kho\u1ea3ng 2-3 gi\u1edd sau khi d\u00f9ng. Khi s\u1eed d\u1ee5ng sau b\u1eefa \u0103n nhi\u1ec1u ch\u1ea5t b\u00e9o l\u00e0m gi\u1ea3m Cmax kho\u1ea3ng 20%<\/span><\/p>\n

              Ph\u00e2n b\u1ed1: Sau khi u\u1ed1ng, th\u1ec3 t\u00edch ph\u00e2n b\u1ed1 (Vd) \u1edf nh\u1eefng \u0111\u1ed1i t\u01b0\u1ee3ng tr\u01b0\u1edfng th\u00e0nh kh\u1ecfe m\u1ea1nh l\u00e0 kho\u1ea3ng 80L. Protein li\u00ean k\u1ebft v\u1edbi huy\u1ebft t\u01b0\u01a1ng c\u1ee7a con ng\u01b0\u1eddi l\u00e0 \u00edt h\u01a1n 6%<\/span><\/p>\n

              Chuy\u1ec3n h\u00f3a: Trofinetide kh\u00f4ng \u0111\u01b0\u1ee3c chuy\u1ec3n h\u00f3a \u0111\u00e1ng k\u1ec3 b\u1edfi c\u00e1c enzym CYP450<\/span><\/p>\n

              Th\u1ea3i tr\u1eeb: Trofinetide \u0111\u01b0\u1ee3c b\u00e0i ti\u1ebft ch\u1ee7 y\u1ebfu qua n\u01b0\u1edbc ti\u1ec3u, v\u1edbi m\u1ed9t l\u01b0\u1ee3ng nh\u1ecf b\u00e0i ti\u1ebft qua ph\u00e2n. Th\u1eddi gian b\u00e1n th\u1ea3i (T1\/2)\u00a0 hi\u1ec7u qu\u1ea3 d\u00f9ng \u0111\u01b0\u1eddng u\u1ed1ng \u1edf nh\u1eefng \u0111\u1ed1i t\u01b0\u1ee3ng kh\u1ecfe m\u1ea1nh l\u00e0 kho\u1ea3ng 1,5 gi\u1edd<\/span><\/p>\n

              2.4 T\u00e1c d\u1ee5ng ph\u1ee5\u00a0<\/span><\/h3>\n

              B\u00ean c\u1ea1nh t\u00e1c d\u1ee5ng c\u1ea7n thi\u1ebft, thu\u1ed1c c\u00f3 th\u1ec3 g\u00e2y ra m\u1ed9t s\u1ed1 t\u00e1c d\u1ee5ng kh\u00f4ng mong mu\u1ed1n nh\u01b0: lo l\u1eafng, gi\u1ea3m s\u1ef1 th\u00e8m \u0103n, b\u1ec7nh ti\u00eau ch\u1ea3y, co gi\u1eadt, gi\u1ea3m c\u00e2n, s\u1ed1t, \u0111au \u0111\u1ea7u, \u0111au c\u01a1, \u0111au h\u1ecdng, ngh\u1eb9t m\u0169i, m\u1ec7t m\u1ecfi,…<\/span><\/p>\n

              2.5 C\u1ea3nh b\u00e1o v\u00e0 th\u1eadn tr\u1ecdng\u00a0<\/span><\/h3>\n

              Ti\u00eau ch\u1ea3y: \u1ede nh\u1eefng b\u1ec7nh nh\u00e2n m\u1eafc h\u1ed9i ch\u1ee9ng Rett \u0111\u01b0\u1ee3c \u0111i\u1ec1u tr\u1ecb b\u1eb1ng Trofinetide, 49% b\u1ecb ti\u00eau ch\u1ea3y k\u00e9o d\u00e0i ho\u1eb7c t\u00e1i ph\u00e1t. M\u1ee9c \u0111\u1ed9 nghi\u00eam tr\u1ecdng c\u1ee7a b\u1ec7nh ti\u00eau ch\u1ea3y l\u00e0 nh\u1eb9 ho\u1eb7c trung b\u00ecnh trong 96% tr\u01b0\u1eddng h\u1ee3p. Ng\u1eebng ho\u1eb7c gi\u1ea3m li\u1ec1u n\u1ebfu x\u1ea3y ra ti\u00eau ch\u1ea3y n\u1eb7ng ho\u1eb7c n\u1ebfu nghi ng\u1edd m\u1ea5t n\u01b0\u1edbc<\/span><\/p>\n

              Gi\u1ea3m c\u00e2n: Trong c\u00e1c nghi\u00ean c\u1ee9u d\u00e0i h\u1ea1n, 2,2% b\u1ec7nh nh\u00e2n ng\u1eebng \u0111i\u1ec1u tr\u1ecb v\u1edbi Daybue do gi\u1ea3m c\u00e2n. Theo d\u00f5i c\u00e2n n\u1eb7ng \u0111\u1ec3 k\u1ecbp th\u1eddi gi\u1ea3m li\u1ec1u ho\u1eb7c ng\u1eebng s\u1eed d\u1ee5ng n\u1ebfu t\u00ecnh tr\u1ea1ng t\u1ec7 h\u01a1n x\u1ea3y ra<\/span><\/p>\n

              3. K\u1ebft lu\u1eadn\u00a0<\/strong><\/h2>\n

              Trofinetide \u0111\u01b0\u1ee3c ch\u1ee9ng minh t\u00ednh hi\u1ec7u qu\u1ea3 cao trong \u0111i\u1ec1u tr\u1ecb \u1edf c\u00e1c b\u1ec7nh nh\u00e2n m\u1eafc ph\u1ea3i h\u1ed9i ch\u1ee9ng Rett v\u1edbi hi\u1ec7u qu\u1ea3 an to\u00e0n t\u01b0\u01a1ng \u0111\u1ed1i cao v\u00e0 mang l\u1ea1i hy v\u1ecdng \u1edf nhi\u1ec1u b\u1ec7nh nh\u00e2n v\u00e0 gia \u0111\u00ecnh h\u1ecd.<\/p>\n","protected":false},"excerpt":{"rendered":"

              Trofinetide l\u00e0 m\u1ed9t lo\u1ea1i thu\u1ed1c \u0111ang \u0111\u01b0\u1ee3c nghi\u00ean c\u1ee9u v\u00e0 s\u1eed d\u1ee5ng trong \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Rett, m\u1ed9t b\u1ec7nh hi\u1ebfm li\u00ean quan \u0111\u1ebfn s\u1ef1 ph\u00e1t tri\u1ec3n n\u00e3o b\u1ed9 \u1edf tr\u1ebb em g\u00e1i. \u0110\u00e2y l\u00e0 ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ph\u00ea duy\u1ec7t cho b\u1ec7nh n\u00e0y v\u00e0 \u0111\u00e3 \u0111\u01b0\u1ee3c FDA (C\u1ee5c qu\u1ea3n l\u00fd Th\u1ef1c […]<\/p>\n","protected":false},"author":54,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":[],"categories":[12],"tags":[],"yoast_head":"\nTrofinetide - Ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean cho h\u1ed9i ch\u1ee9ng Rett<\/title>\n<meta name=\"description\" content=\"Trofinetide l\u00e0 m\u1ed9t ph\u01b0\u01a1ng ph\u00e1p \u0111i\u1ec1u tr\u1ecb \u0111\u1ea7u ti\u00ean \u0111\u01b0\u1ee3c ch\u1ea5p thu\u1eadn v\u00e0 \u0111\u01b0\u1ee3c s\u1eed dugnj trong \u0111i\u1ec1u tr\u1ecb h\u1ed9i ch\u1ee9ng Rett\" \/>\n<meta name=\"robots\" content=\"noindex, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Trofinetide - 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